Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorder
Abstract Autism Spectrum Disorder (ASD) is a neurodevelopmental disorder characterized by core impairments in social communication as well as restricted, repetitive patterns of behavior and/or interests. Individuals with ASD, which includes about 2% of the US population, have challenges with activit...
| Main Authors: | , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Nature Portfolio
2023-03-01
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| Series: | Scientific Reports |
| Online Access: | https://doi.org/10.1038/s41598-023-30909-0 |
| _version_ | 1827974546452381696 |
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| author | Lauren M. Schmitt Elizabeth G. Smith Ernest V. Pedapati Paul S. Horn Meredith Will Martine Lamy Lillian Barber Joe Trebley Kevin Meyer Mark Heiman Korbin H. J. West Phoevos Hughes Sanjeev Ahuja Craig A. Erickson |
| author_facet | Lauren M. Schmitt Elizabeth G. Smith Ernest V. Pedapati Paul S. Horn Meredith Will Martine Lamy Lillian Barber Joe Trebley Kevin Meyer Mark Heiman Korbin H. J. West Phoevos Hughes Sanjeev Ahuja Craig A. Erickson |
| author_sort | Lauren M. Schmitt |
| collection | DOAJ |
| description | Abstract Autism Spectrum Disorder (ASD) is a neurodevelopmental disorder characterized by core impairments in social communication as well as restricted, repetitive patterns of behavior and/or interests. Individuals with ASD, which includes about 2% of the US population, have challenges with activities of daily living and suffer from comorbid medical and mental health concerns. There are no drugs indicated for the core impairments of ASD. As such, there is a significant need for the development of new medication strategies for individuals with ASD. This first-in-human placebo-controlled, double-blind, crossover study investigated the safety (primary objective) and efficacy of oral SB-121, a combination of L. reuteri, Sephadex® (dextran microparticles), and maltose administered once daily for 28 days in 15 autistic participants. SB-121 was safe and well tolerated. SB-121-associated directional improvements in adaptive behavior measured by Vineland-3 and social preference as measured with eye tracking were noted. These results provide support for further clinical evaluation of SB-121 as a treatment in autistic patients. To evaluate the safety and tolerability of multiple doses of SB-121 in subjects with autism spectrum disorder. Single-center, randomized, placebo-controlled, double-blind, crossover trial. 15 patients with autism spectrum disorder were randomized and analyzed. Daily dosing of SB-121 or placebo for 28 days, followed by approximately a 14 day washout, then 28 days of dosing with other treatment. Incidence and severity of adverse events, presence of Limosilactobacillus reuteri and Sephadex® in stool, and incidence of bacteremia with positive L. reuteri identification. Additional outcomes include changes from baseline on cognitive and behavior tests as well as biomarker levels. Adverse event rates were similar between SB-121 and placebo, with most reported as mild. There were no severe or serious adverse events. No participants had features of suspected bacteremia or notable changes in vital signs, safety laboratory, or ECG parameters from baseline. There was a statistically significant increase from baseline in the Vineland-3 Adaptive Behavior Composite score (p = 0.03) during SB-121 treatment. There was a trend for increased social/geometric viewing ratio following SB-121 treatment compared to placebo. SB-121 was safe and well tolerated. SB-121-associated directional improvements in adaptive behavior measured by Vineland-3 and social preference as measured with eye tracking were noted. Trial registration: clinicaltrials.gov Identifier: NCT04944901. |
| first_indexed | 2024-04-09T19:56:05Z |
| format | Article |
| id | doaj.art-2770a27e6a904544bc655818432bdc53 |
| institution | Directory Open Access Journal |
| issn | 2045-2322 |
| language | English |
| last_indexed | 2024-04-09T19:56:05Z |
| publishDate | 2023-03-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Scientific Reports |
| spelling | doaj.art-2770a27e6a904544bc655818432bdc532023-04-03T05:26:38ZengNature PortfolioScientific Reports2045-23222023-03-0113111210.1038/s41598-023-30909-0Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorderLauren M. Schmitt0Elizabeth G. Smith1Ernest V. Pedapati2Paul S. Horn3Meredith Will4Martine Lamy5Lillian Barber6Joe Trebley7Kevin Meyer8Mark Heiman9Korbin H. J. West10Phoevos Hughes11Sanjeev Ahuja12Craig A. Erickson13Division of Behavioral Medicine and Clinical Psychology, Cincinnati Children’s Hospital Medical CenterDivision of Behavioral Medicine and Clinical Psychology, Cincinnati Children’s Hospital Medical CenterDivision of Child and Adolescent Psychiatry, Cincinnati Children’s Hospital Medical CenterDepartment of Pediatrics, University of Cincinnati College of MedicineDivision of Behavioral Medicine and Clinical Psychology, Cincinnati Children’s Hospital Medical CenterDivision of Child and Adolescent Psychiatry, Cincinnati Children’s Hospital Medical CenterDivision of Child and Adolescent Psychiatry, Cincinnati Children’s Hospital Medical CenterScioto Biosciences, Inc.Scioto Biosciences, Inc.Scioto Biosciences, Inc.Scioto Biosciences, Inc.Scioto Biosciences, Inc.Scioto Biosciences, Inc.Division of Child and Adolescent Psychiatry, Cincinnati Children’s Hospital Medical CenterAbstract Autism Spectrum Disorder (ASD) is a neurodevelopmental disorder characterized by core impairments in social communication as well as restricted, repetitive patterns of behavior and/or interests. Individuals with ASD, which includes about 2% of the US population, have challenges with activities of daily living and suffer from comorbid medical and mental health concerns. There are no drugs indicated for the core impairments of ASD. As such, there is a significant need for the development of new medication strategies for individuals with ASD. This first-in-human placebo-controlled, double-blind, crossover study investigated the safety (primary objective) and efficacy of oral SB-121, a combination of L. reuteri, Sephadex® (dextran microparticles), and maltose administered once daily for 28 days in 15 autistic participants. SB-121 was safe and well tolerated. SB-121-associated directional improvements in adaptive behavior measured by Vineland-3 and social preference as measured with eye tracking were noted. These results provide support for further clinical evaluation of SB-121 as a treatment in autistic patients. To evaluate the safety and tolerability of multiple doses of SB-121 in subjects with autism spectrum disorder. Single-center, randomized, placebo-controlled, double-blind, crossover trial. 15 patients with autism spectrum disorder were randomized and analyzed. Daily dosing of SB-121 or placebo for 28 days, followed by approximately a 14 day washout, then 28 days of dosing with other treatment. Incidence and severity of adverse events, presence of Limosilactobacillus reuteri and Sephadex® in stool, and incidence of bacteremia with positive L. reuteri identification. Additional outcomes include changes from baseline on cognitive and behavior tests as well as biomarker levels. Adverse event rates were similar between SB-121 and placebo, with most reported as mild. There were no severe or serious adverse events. No participants had features of suspected bacteremia or notable changes in vital signs, safety laboratory, or ECG parameters from baseline. There was a statistically significant increase from baseline in the Vineland-3 Adaptive Behavior Composite score (p = 0.03) during SB-121 treatment. There was a trend for increased social/geometric viewing ratio following SB-121 treatment compared to placebo. SB-121 was safe and well tolerated. SB-121-associated directional improvements in adaptive behavior measured by Vineland-3 and social preference as measured with eye tracking were noted. Trial registration: clinicaltrials.gov Identifier: NCT04944901.https://doi.org/10.1038/s41598-023-30909-0 |
| spellingShingle | Lauren M. Schmitt Elizabeth G. Smith Ernest V. Pedapati Paul S. Horn Meredith Will Martine Lamy Lillian Barber Joe Trebley Kevin Meyer Mark Heiman Korbin H. J. West Phoevos Hughes Sanjeev Ahuja Craig A. Erickson Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorder Scientific Reports |
| title | Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorder |
| title_full | Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorder |
| title_fullStr | Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorder |
| title_full_unstemmed | Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorder |
| title_short | Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorder |
| title_sort | results of a phase ib study of sb 121 an investigational probiotic formulation a randomized controlled trial in participants with autism spectrum disorder |
| url | https://doi.org/10.1038/s41598-023-30909-0 |
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